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. 2025 Nov 25;22(2):105–117. doi: 10.1080/14796678.2025.2583805

A real-world study of tafamidis in people with transthyretin amyloid cardiomyopathy (ATTR-CM) with heart and nerve symptoms: a plain language summary

Jonas Wixner a, Angela Dispenzieri b, Leslie Amass c, Martin Carlsson c, Steve Riley d, Evan Powers e, Jeffery W Kelly e
PMCID: PMC12915833  PMID: 41292260

Plain Language Summary

What is this summary about?

This summary describes results from The Transthyretin Amyloidosis Outcomes Survey (THAOS for short). THAOS is a real-world study of people with a heart condition called transthyretin amyloid cardiomyopathy (ATTR-CM for short). Researchers looked at how tafamidis affects people with ATTR-CM who had both heart- and nerve-related symptoms. They calculated how long people lived and compared the data between those who took tafamidis and those who did not. They also compared how long and how often people had to stay in the hospital. Lastly, researchers looked at the side effects that people had during the study when they took tafamidis.

What are the key takeaways?

After two and a half years, a larger proportion of people who took tafamidis (82% or 82 in 100) were estimated to be alive than people who did not take tafamidis (75% or 75 in 100). On average, people who took tafamidis were hospitalized for heart-related problems less often than people who did not take tafamidis. People who took tafamidis also had shorter hospital stays for heart-related problems. The side effects people had when taking tafamidis were similar to those reported in previous clinical studies.

What were the main conclusions reported by the researchers?

The results from this real-world study suggest that people with ATTR-CM with heart- and nerve-related symptoms who take tafamidis may live longer with less frequent and shorter hospital stays for heart-related symptoms.

Clinical trial number: NCT00628745

KEYWORDS: Heart failure, Cardiomyopathies, amyloidosis, transthyretin, real-world outcomes

Acknowledgements

Pfizer thanks everyone who took part in this study.

This is an abstract of the Plain Language Summary of Publication article.

To read the full Plain Language Summary of this article, click here to view the PDF.

Link to original article here

Disclosure statement

Jonas Wixner reports consulting fees and travel support for lectures and advisory boards from Alnylam and Pfizer and consulting fees from Akcea, AstraZeneca, Bayer, and Intellia. Angela Dispenzieri reports research grants from Alnylam, Celgene, Janssen, Millennium, and Pfizer; funding from Pfizer for meeting expenses (travel); and attending advisory boards for Akcea and Intellia. Leslie Amass, Martin Carlsson, and Steve Riley are employees of Pfizer. Evan Powers receives royalty payments from tafamidis sales. Teresa Coelho serves as a medical advisor for Pfizer and reports funding from Pfizer for scientific meeting expenses (travel, accommodations, and registration). Jeffery W Kelly receives royalty payments from tafamidis sales and reports consulting fees and travel support from Pfizer for lectures and attending advisory boards. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

Medical writing support was provided by Emily Balevich, PhD, of Engage Scientific Solutions, and was funded by Pfizer. Authors of the original article were involved in preparing this summary.

Pfizer’s generative artificial intelligence (AI) assisted technology, MAIA (Medical Artificial Intelligence Assistant), was used in the production of this summary to develop the text. After using this tool/service, the authors reviewed and edited the content as needed and take full responsibility for the content of the summary.

Patient reviewers on this PLSP have received honorarium from Future Cardiology for their review work but have no other relevant financial relationships to disclose.

Peer reviewers on this manuscript have no relevant financial or other relationships to disclose.

Funding

This plain language summary was funded by Pfizer. Pfizer contributed to the study design and management and collection of data for the original study. In their role as authors, employees of Pfizer were involved in the preparation, review, and approval of this plain language summary, and the decision to submit it for publication, along with their co-authors.


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